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Sanfilippo Mucopolysaccharidoses clinical trials

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NCT ID: NCT02254863 Recruiting - Clinical trials for Mucopolysaccharidosis II

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

DUOC-01
Start date: September 2014
Phase: Phase 1
Study type: Interventional

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.